Gene Augmentation Therapy Restores Retinal Function and Visual Behavior in a Sheep Model of CNGA3 Achromatopsia
about
Large animal models of rare genetic disorders: sheep as phenotypically relevant models of human genetic diseaseCanine CNGA3 Gene Mutations Provide Novel Insights into Human Achromatopsia-Associated Channelopathies and TreatmentCLINICAL PROGRESS IN INHERITED RETINAL DEGENERATIONS: GENE THERAPY CLINICAL TRIALS AND ADVANCES IN GENETIC SEQUENCING.AAV-Mediated Gene Supplementation Therapy in Achromatopsia Type 2: Preclinical Data on Therapeutic Time Window and Long-Term EffectsResidual Foveal Cone Structure in CNGB3-Associated Achromatopsia.Advances in Gene Therapy for Diseases of the Eye.Gene Augmentation Therapy for a Missense Substitution in the cGMP-Binding Domain of Ovine CNGA3 Gene Restores Vision in Day-Blind Sheep.Animal modelling for inherited central vision loss.Gene and cell-based therapies for inherited retinal disorders: An update.Gene therapy for achromatopsia.Hereditary Retinal Dystrophy.Retinal Gene Therapy: Surgical Vector Delivery in the Translation to Clinical Trials.Development of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE65.Protocols for Visually Guided Navigation Assessment of Efficacy of Retina-Directed Cell or Gene Therapy in CaninesConcepts and Strategies in Retinal Gene Therapy.Long-term retinal cone rescue using a capsid mutant AAV8 vector in a mouse model of CNGA3-achromatopsia.Synthetic adeno-associated viral vector efficiently targets mouse and non-human primate retina in vivo.Achromatopsia: clinical features, molecular genetics, animal models and therapeutic options.Retinal Cyclic Nucleotide-Gated Channels: From Pathophysiology to Therapy.Gene Therapy for Color Blindness.Gene therapy and genome surgery in the retina.Efficient Transduction and Expansion of Ovine Macrophages for Gene Therapy Implementations.Longitudinal In Vivo Monitoring of the CNS Demonstrates the Efficacy of Gene Therapy in a Sheep Model of CLN5 Batten DiseaseCRISPR/Cas9 genome surgery for retinal diseases
P2860
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P2860
Gene Augmentation Therapy Restores Retinal Function and Visual Behavior in a Sheep Model of CNGA3 Achromatopsia
description
2015 nî lūn-bûn
@nan
2015 թուականի Սեպտեմբերին հրատարակուած գիտական յօդուած
@hyw
2015 թվականի սեպտեմբերին հրատարակված գիտական հոդված
@hy
2015年の論文
@ja
2015年論文
@yue
2015年論文
@zh-hant
2015年論文
@zh-hk
2015年論文
@zh-mo
2015年論文
@zh-tw
2015年论文
@wuu
name
Gene Augmentation Therapy Rest ...... p Model of CNGA3 Achromatopsia
@ast
Gene Augmentation Therapy Rest ...... p Model of CNGA3 Achromatopsia
@en
Gene Augmentation Therapy Rest ...... p Model of CNGA3 Achromatopsia
@nl
type
label
Gene Augmentation Therapy Rest ...... p Model of CNGA3 Achromatopsia
@ast
Gene Augmentation Therapy Rest ...... p Model of CNGA3 Achromatopsia
@en
Gene Augmentation Therapy Rest ...... p Model of CNGA3 Achromatopsia
@nl
prefLabel
Gene Augmentation Therapy Rest ...... p Model of CNGA3 Achromatopsia
@ast
Gene Augmentation Therapy Rest ...... p Model of CNGA3 Achromatopsia
@en
Gene Augmentation Therapy Rest ...... p Model of CNGA3 Achromatopsia
@nl
P2093
P2860
P3181
P356
P1433
P1476
Gene Augmentation Therapy Rest ...... p Model of CNGA3 Achromatopsia
@en
P2093
Alexander Rosov
Alexey Obolensky
Ayala Ejzenberg
Dror Sharon
Edward Averbukh
Elisha Gootwine
Esther Yamin
Eyal Banin
Lina Zelinger
P2860
P304
P3181
P356
10.1038/MT.2015.114
P407
P577
2015-06-19T00:00:00Z